AAVantgarde Bio, an Italian-based biotechnology company, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to AAVB-081, a gene therapy being developed for the treatment of Usher Syndrome Type 1B (USH1B) retinitis pigmentosa. This designation highlights the urgent need for effective therapies for this rare, inherited condition that leads to progressive vision loss and blindness.
Significance of Orphan Drug Designation
The FDA's Orphan Drug Designation program is designed to support the development of drugs and biologics for rare diseases. It provides several key incentives, including tax credits for qualified clinical trials, exemption from FDA user fees, and the potential for seven years of market exclusivity following FDA approval. These incentives are intended to encourage pharmaceutical companies to invest in developing treatments for conditions that affect relatively small patient populations.
AAVB-081: A Novel Gene Therapy Approach
AAVB-081 is an intra-retinal Adeno-Associated Viral (AAV) vector-based therapy that targets Usher Syndrome Type 1B, which is associated with mutations in the MYO7A gene. AAVantgarde's dual hybrid platform uses two AAV8 vectors, each containing one half of an expression cassette encoding for the Myo7A gene. These vectors work at the cell nucleus level, recombining the two halves of the transgene back into a single one within the cell. This approach is designed to overcome the cargo capacity limitations of standard AAV vectors, as the MYO7A gene is 6.7 kb long, making it too large for a single AAV vector.
Usher Syndrome Type 1B: An Unmet Medical Need
Usher Syndrome Type 1B is a rare, inherited disorder characterized by deafness from birth, vestibular dysfunction, and progressive vision loss due to retinitis pigmentosa. It is caused by mutations in the MYO7A gene. Approximately 20,000 patients in the U.S. and E.U. are affected by Usher Syndrome Type 1B. While surgical treatments exist to address the deafness associated with the condition, there are currently no approved therapies to prevent or reverse the progressive vision loss.
Executive Perspective
"People living with USH1B are in urgent need of a therapy to prevent their progressive loss of vision leading to eventual blindness. Receiving FDA Orphan Drug Designation reinforces the unmet need of this indication and is an important milestone in the development of this program," said Dr. Natalia Misciattelli, Chief Executive Officer of AAVantgarde.
